Sat.Jun 17, 2023 - Fri.Jun 23, 2023

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First Duchenne gene therapy approved by FDA for young children

BioPharma Drive: Drug Pricing

The conditional clearance for Sarepta’s Elevidys in 4- and 5-year-olds is a milestone for research into the deadly disease, and raises the stakes of an ongoing trial that could prove how well it works.

Therapies 130
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AHA News: Beat the Heat With Hydrating Foods This Summer

Drugs.com

TUESDAY, June 20, 2023 (American Heart Association News) -- Staying hydrated can help you live a longer, healthier life. But drinking multiple glasses of water throughout the day isn't for everyone. Health experts say that's OK. While fluids are.

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Clean, sustainable fuels made 'from thin air' and plastic waste

Science Daily: Pharmacology News

Researchers have demonstrated how carbon dioxide can be captured from industrial processes -- or even directly from the air -- and transformed into clean, sustainable fuels using just the energy from the Sun.

Research 117
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Novel Oncology Target Claudin 6 Makes its Case in Solid Tumors

Fierce BioTech

Harnessing the Power Within | In the ongoing search for new cancer treatments, companies including Context Therapeutics are developing potential therapies focused on the emerging novel oncology target Claudin 6.

Therapies 111
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From Diagnosis to Delivery: How AI is Revolutionizing the Patient Experience

Speaker: Simran Kaur, Founder & CEO at Tattva Health Inc.

The healthcare landscape is being revolutionized by AI and cutting-edge digital technologies, reshaping how patients receive care and interact with providers. In this webinar led by Simran Kaur, we will explore how AI-driven solutions are enhancing patient communication, improving care quality, and empowering preventive and predictive medicine. You'll also learn how AI is streamlining healthcare processes, helping providers offer more efficient, personalized care and enabling faster, data-driven

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UniQure stock tumbles on ‘confusing’ results for Huntington’s gene therapy

BioPharma Drive: Drug Pricing

While treated patients appear to be doing better than history suggests they would, mixed findings involving a key surrogate marker perplexed Wall Street analysts and sent shares falling 38%.

Therapies 120
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When Your Child Has Scoliosis: Symptoms & Treatments

Drugs.com

FRIDAY, June 23, 2023 -- The future for a child diagnosed with scoliosis, an abnormal curvature of the spine that typically occurs just before puberty, can feel uncertain. As a parent, you want answers to your questions about scoliosis and how it.

Treatment 119

More Trending

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Do hummingbirds drink alcohol? More often than you think

Science Daily: Pharmacology News

Animals that eat fruit or sip nectar often ingest alcohol because naturally occurring yeasts turning sugar into ethanol. But how do animals feel about that? A new study details an experiment to determine whether hummingbirds are turned off by alcohol in sugar water. At 1% by volume, no. At 2% by volume, they consume much less. The implication is that hummingbirds have adjusted to small amounts of alcohol likely present in flowers and backyard feeders.

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Sarepta prices Duchenne gene therapy at $3.2M

BioPharma Drive: Drug Pricing

The cost makes Sarepta’s treatment, called Elevidys, among the most expensive medicines in the world. But company executives said they don’t expect significant insurer pushback.

Therapies 117
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Legalizing Marijuana Tied to More Binge Drinking in Folks Over 30

Drugs.com

WEDNESDAY, June 21, 2023 -- A growing number of states are legalizing marijuana for recreational use, and it may be leading to an unexpected side effect among millennials and Gen Xers: binge drinking. Binge drinking is defined as five or more drinks.

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International Women in Engineering Day

NIOSH Science Blog: Drugs

June 23, 2023 is International Women in Engineering Day. In the United States, women make up 14% of the engineering workforce (15% internationally).[1] In 2020, 24% of bachelor’s degrees in engineering were earned by women, and women of color earned 10% of the total engineering degrees.[1] The National Institute for Occupational Safety and Health (NIOSH) understands the importance of diversity throughout the STEM fields and in occupational safety and health.

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How Machine Learning Drives Clinical Trial Efficiency

Clinical trial data management is increasingly challenging as studies grow in complexity. Quickly accessing and analyzing study data is vital for assessing trial progress and patient safety. In this paper, we explore real-time data access and analysis for proactive study management. We investigate using adverse event (AE) data to monitor safety and discuss a clinical analytics platform that supports collaboration and data review workflows.

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Low oxygen levels restore balance and coordination in a mouse model of a movement disorder

Broad Institute

Low oxygen levels restore balance and coordination in a mouse model of a movement disorder By Allessandra DiCorato June 21, 2023 Breadcrumb Home Low oxygen levels restore balance and coordination in a mouse model of a movement disorder New study suggests that treatments that mimic the effects of a low-oxygen environment could one day help reverse Friedreich’s ataxia.

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Flagship’s latest startup aims to take the guesswork out of small molecule drugs

BioPharma Drive: Drug Pricing

Empress Therapeutics claims its technology allows it to identify chemical drug candidates faster and more reliably. It has $50 million from Flagship to prove it can.

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FDA Orders Stores to Stop Selling Elf Bar Disposable Vapes

Drugs.com

FRIDAY, June 23, 2023 -- The U.S. Food and Drug Administration said Thursday it has sent warning letters to 189 retailers, telling them to stop selling unauthorized tobacco products such as Elf Bar and Esco Bars. Both brands are disposable.

FDA 119
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#ScienceSaturday: June 17, 2023

KIF1A

#ScienceSaturday posts share exciting scientific developments and educational resources with the KAND community. Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. KIF1A-Related Research KIF1A neurodegenerative disease mutations modulate motor motility and force generation One challenge in researching KAND is that a patient’s symptoms can vary so much depending on the type

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Deliver Fast, Flexible Clinical Trial Insights with Spotfire

Clinical research has entered a new era, one that requires real-time analytics and visualization to allow trial leaders to work collaboratively and to develop, at the click of a mouse, deep insights that enable proactive study management. Learn how Revvity Signals helps drug developers deliver clinical trial data insights in real-time using a fast and flexible data and analytics platform to empower data-driven decision-making.

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Optimizely CMS – Getting all content of a specific property with a simple SQL script

Perficient: Drug Development

When you need to retrieve all content of a specific property from a Page/Block type, normally you will use the IContentLoader or IContentRepository, or even IContentModelUsage to get all instances of a content type then select them by property. This is the correct implementation by code. But what if you only need to check the property’s value for debugging/development purpose?

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Patient death spurs FDA to pause test of Arcellx’s Gilead-partnered cell therapy

BioPharma Drive: Drug Pricing

The company said limitations on "bridging treatment" used to hold cancer in check could be to blame, and is working with the FDA to change the study's protocol.

FDA 107
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Cirrhosis of the Liver: Causes, Symptoms & Treatment

Drugs.com

TUESDAY, June 20, 2023 -- Cirrhosis of the liver poses a significant health challenge worldwide. With no known cure, it requires comprehensive management to slow liver damage, alleviate symptoms and prevent potential complications. Cirrhosis is.

Treatment 118
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Combination therapy effective against canine melanoma

SCIENMAG: Medicine & Health

A combination of radiotherapy followed by immunotherapy is a promising strategy for the treatment of oral malignant melanomas in dogs. Credit: Tatsuya Deguchi, Naoya Maekawa, et al. Cancers. June 1, 2023 A combination of radiotherapy followed by immunotherapy is a promising strategy for the treatment of oral malignant melanomas in dogs.

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Clinical Data Like You´ve Never Seen It Before: Why Spotfire Is the Leading Tool for Clinical Analytics

Clinical development organizations face a wide array of challenges when it comes to data, many of which can impact the operational effectiveness of their clinical trials. In this whitepaper, experts from Revvity Signals explore how solutions like TIBCO® Spotfire® enable better, more streamlined studies. The whitepaper also features a success story from Ambrx, a leading biopharmaceutical company, detailing how it has leveraged Spotfire to tackle data quality and collaboration challenges in clinic

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7 Reasons Projects Fail (And How to Ensure Success)

thought leadership

Leadership at drug and devices companies are continually overwhelmed with the constant balance of driving daily work, managing resources, and addressing unplanned emergencies. This balance is then tested when leadership is tasked with implementing a number of yearly special projects. These projects often address process, strategic, technical, or regulatory requirements that must be addressed within a specific timeframe.

Drugs 89
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Top FDA official overruled review team in approval of Sarepta’s Duchenne gene therapy

BioPharma Drive: Drug Pricing

In a memo made public Friday, Peter Marks, head of the agency office that oversees gene therapies, wrote that he disagreed with other reviewers and saw “compelling” evidence to clear Elevidys.

Therapies 103
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Is Child's Tummy Pain a Serious Concern? Poll Finds Many Parents Unsure

Drugs.com

MONDAY, June 19, 2023 -- Kids get a lot of tummy aches. How a parent responds to it can vary, just as the causes can, according to the University of Michigan Health's C.S. Mott Children’s Hospital National Poll on Children’s Health, which looked.

Hospitals 118
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Sinking seamount offers clues to slow motion earthquakes

SCIENMAG: Medicine & Health

Scientists have long puzzled over what happens when seamounts — mountains and volcanoes on the seafloor — are pulled into subduction zones. Now, new research from The University of Texas at Austin shows that when seamounts sink, they leave behind a trail of soft sediments.

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How to use Zebrafish for Cannabinoid studies

biobide

Cannabinoids are compounds that target the cannabinoid receptors. These types of alkaloid compounds come, mostly, from Cannabis genus plants, even though can be of synthetic or semisynthetic nature. They include psychoactive and non-psychoactive active ingredients. Tetrahydrocannabinol (THC) is the main psychoactive compound, and its psychotropic effects are of clinical concern as it may heighten anxiety and can potentially cause addiction and cravings.

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Medicare has no surprises in planned registry for new Alzheimer’s drugs

BioPharma Drive: Drug Pricing

Ahead of Leqembi’s possible full approval next month, the agency released details emphasizing how the required registry to track patient outcomes will be free and easy to use.

Drugs 103
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FDA Approves First Gene Therapy, Elevidys, to Treat Duchenne Muscular Dystrophy

Drugs.com

FRIDAY, June 23, 2023 -- The U.S. Food and Drug Administration on Thursday approved the drug Elevidys, the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD).

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Brain pathway identified that impairs postpartum social behavior after adolescent stress

SCIENMAG: Medicine & Health

BIRMINGHAM, Ala. – Stress during adolescence can cause postpartum behavioral changes in women and other mammals, including depression and changes in social behavior after the birth of a child. Credit: UAB BIRMINGHAM, Ala.

Science 93
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Getting to Know Expansion Microscopy

addgene Blog

Expansion Microscopy (ExM) promises an easier, more accessible way to image biological features previously only visible via techniques like super-resolution microscopy or electron microscopy. Since its introduction in 2015 by the Boyden Lab , ExM has been steadily growing in popularity with an abundance of protocols and examples of applications from across disciplines now available.

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Cell and gene therapy manufacturing: the next generation of startups

BioPharma Drive: Drug Pricing

Joining larger CDMOs like Lonza and Catalent, a new group of companies aims to capitalize on persistent bottlenecks in producing complex genetic treatments.

Therapies 111
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Screen All Adults Under Age 65 for Anxiety Disorders, Expert Panel Says

Drugs.com

TUESDAY, June 20, 2023 -- For the first time ever, the nation’s top panel of preventive health experts has recommended that doctors routinely screen all adult patients under 65 for anxiety disorder. Evidence now shows that anxiety screening can.

Doctors 111
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Forensic bitemark analysis for court trials is not supported by sufficient data and “is leading to wrongful convictions”

SCIENMAG: Medicine & Health

The commonly-used evidence in trials, bitemark analysis, is not backed up by scientific research – an analysis of current literature, and 12 new studies, shows.

Trials 97
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New study reveals global reservoirs are becoming emptier

Science Daily: Pharmacology News

Over the past two decades, global reservoirs have become increasingly empty despite an overall increase in total storage capacity due to the construction of new reservoirs. Researchers used a new approach with satellite data to estimate the storage variations of 7,245 global reservoirs from 1999 to 2018.

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Bluebird, playing catch-up, gets decision date for sickle cell gene therapy

BioPharma Drive: Drug Pricing

The regulator will issue a decision on lovo-cel by Dec. 20, roughly two weeks after a verdict is expected on a rival gene editing treatment from Vertex and CRISPR Therapeutics.

Therapies 100
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Pancreatic Neuroendocrine Tumors: Slow-Growing and Often Curable

Drugs.com

MONDAY, June 19, 2023 -- Pancreatic cancer is often considered a death sentence, typically aggressive and usually caught late, but there is one type of pancreatic cancer that is far more treatable. While over 90% of pancreatic cancers are.

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